Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers
Last verified by NonDilute: 2026-06-08. Official notice and agency instructions control.
Build infrastructure or novel methods to overcome the clinical, regulatory, and enrollment barriers blocking treatments for cancers that affect fewer than 400 Americans annually.
Report stale or inaccurate summary
What this is
This FDA Oncology Center of Excellence funding targets the specific bottlenecks in ultra-rare cancer drug development: patient recruitment, lack of clinical endpoints, limited disease understanding, and poor financial incentives. Applicants can propose coordination networks, real-world data registries, repurposing of discontinued drugs, pragmatic trial designs, or novel delivery/characterization approaches for cancers with ≤400 annual cases. Awards up to $500,000 support both infrastructure and pre-clinical/early-clinical research.
Who can apply
For-profit small businesses, nonprofits (501(c)(3) and non-501(c)(3)), universities, government entities, tribal organizations, and other institution types are eligible. No explicit geographic restriction; domestic focus implied by FDA jurisdiction. No size cap on for-profits or nonprofits (beyond small-business definition for SBs).
Eligible applicant types
- State governments
- Public and State controlled institutions of higher education
- Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
- Special district governments
- Independent school districts
- Private institutions of higher education
- County governments
- For profit organizations other than small businesses
- Small businesses
- Public housing authorities/Indian housing authorities
- Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
- Native American tribal governments (Federally recognized)
- Native American tribal organizations (other than Federally recognized tribal governments)
- City or township governments
Full description — from the agency
Background The FDA Oncology Center of Excellence (OCE) aims to advance the development and regulation of oncology products for patients with cancer. The Pediatric Oncology Program and Rare Cancers Program were established to facilitate and expedite drug development for pediatric and other rare cancers. OCE’s Project Catalyst connects scientific knowledge, creative insight, and medical professionals to foster early-stage product innovation, which is particularly important to address the challenges related to product development for ultra-rare cancers. In collaboration with the National Institutes of Health (NIH) and the OCE, the Foundation for the National Institutes of Health (FNIH) recently launched the design phase of the Ultra-Rare Cancer Treatment Advancement Program (ULTRA), a new public-private partnership dedicated to accelerating the development of innovative ultra-rare cancer treatments. For the purposes of this NOFO, the FDA OCE refers to cancers with an approximate annual incidence in the U.S. of 300 to 400 people or less as ultra-rare (a more stringent criterion compared to the threshold for a rare disease specified in the Orphan Drug Act based on a U.S. prevalence of Many of the challenges involved in drug development for ultra-rare cancers are similar to those for rare diseases and can include: Difficulty enrolling sufficient numbers of patients to clinical trials Limited financial incentives for drug development Insufficient understanding of the cancer pathophysiology, molecular characteristics, and natural history Limited or lack of timely access to molecular testing to determine eligibility for treatment with targeted therapies Complexities associated with designing clinical trials that are adequate to establish safety and effectiveness Advancing technologies such as single cell multi-omic analyses have helped define some ultra-rare cancers at the molecular level, providing new opportunities for targeted drug development. Pediatric oncology has several examples of tumor types with known translocation-induced, oncogenic driver fusion proteins. Other examples of ultra-rare cancers defined today by molecular pathology include: neuroectodermal tumors, pulmonary blastoma, desmoplastic small round cell tumor (DSRCT), epithelioid sarcoma, diffuse intrinsic pontine glioma, fibrolamellar carcinoma, and malignant rhabdoid tumors. Purpose and Research Objectives The purpose of this NOFO is to support new approaches that can be applied to facilitate therapeutic development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. Specific areas of interest include, but are not limited to, the following examples: Development of infrastructure for a coordination network and data repository for patient-level data across institutions and internationally to support drug development and regulatory decision-making for one or more ultra-rare cancers. Investigations to explore opportunities to develop and validate early clinical endpoints and other novel efficacy endpoints for evaluation of treatments for ultra-rare cancers. Development and implementation of a collaborative multi-stakeholder effort to support generation and use of real-world data leveraging a registry framework for use in development of new therapies for pediatric patients with diffuse midline glioma (DMG) (including diffuse intrinsic pontine glioma, DIPG). Innovative approaches to identify new biologically-driven opportunities for clinical development of previously approved drugs or biologics (hereafter referred to as drugs), including drugs for which development has been discontinued, in ultra-rare cancers. Research to develop novel approaches to preserve the availability of drugs for which commercial developers have discontinued adult development that have strong potential in ultra-rare cancers but lack financial incentives for commercial development Development of methods to incorpo…
Topics: ultra-rare cancer drug development · pediatric oncology · clinical endpoints · patient registry · drug repurposing · rare disease · therapeutic advancement
Public-source funding discovery only. This summary is generated from public agency data and may be incomplete or stale. NonDilute is not affiliated with, endorsed by, or acting on behalf of any government agency. Official notices and agency instructions control. NonDilute does not determine eligibility, provide grant-writing advice, or guarantee funding.